Shape Medical Strategy for a Leading Rare Disease Portfolio
We are partnering with a global biotechnology company recognized for building one of the most respected rare disease franchises in the industry. The organization is deeply science-driven, patient-centric, and known for advancing therapies that address serious, genetically driven, and life-limiting conditions where treatment options have historically been limited or nonexistent.
The company has a robust and diversified rare disease pipeline, spanning:
- Commercially established therapies with long-term real-world data
- Late-stage clinical programs approaching pivotal or registration milestones
- Early- to mid-stage assets leveraging advanced modalities and novel mechanisms of action
As the portfolio expands across multiple rare indications and geographies, the organization is seeking a Senior Director, Medical Affairs to provide strategic leadership across medical strategy, evidence generation, and external engagement.
The Opportunity
This role sits at the heart of the organization’s rare disease strategy. As Senior Director, Medical Affairs, you will be responsible for shaping how complex scientific data are translated into credible, patient-meaningful, and regulator- and payer-relevant medical narratives.
You will work across the full product lifecycle—from late-stage development through commercialization and lifecycle expansion—and will be a key partner to Clinical Development, Regulatory Affairs, Market Access, and Commercial leadership. The role offers both strategic influence and hands-on impact, with visibility at the highest levels of the organization.
About the Organization & Portfolio
- Global footprint with strong U.S. and international presence
- Established leadership in genetic and rare diseases, with expanding investment into adjacent rare and specialty indications
- Portfolio includes therapies requiring:
- Long-term follow-up and durability assessment
- Sophisticated benefit–risk communication
- Deep engagement with patient advocacy communities
- Pipeline reflects a mix of small molecules, advanced biologics, and next-generation modalities, requiring strong medical governance and evidence planning
- Organization is known for close collaboration with regulators and for setting new benchmarks in rare disease development and commercialization
Key Responsibilities
Medical Strategy & Franchise Leadership
- Lead the development and execution of global and/or U.S. medical affairs strategy across one or more rare disease franchises
- Serve as a senior medical leader on asset, franchise, and lifecycle teams, influencing strategic decisions from late-stage development through post-approval
- Ensure medical strategy evolves in step with emerging data, competitive landscape, and patient needs
Evidence Generation & Scientific Leadership
- Oversee integrated evidence planning, including publications, real-world evidence, registries, observational studies, and long-term follow-up strategies
- Guide the development of medical narratives that clearly articulate disease burden, treatment value, durability, and patient impact
- Provide senior oversight of scientific communications, congress strategy, and peer-reviewed publications
External Engagement & Advocacy
- Build trusted relationships with KOLs, investigators, and external experts across rare disease communities
- Partner closely with patient advocacy organizations, ensuring patient voice is meaningfully incorporated into strategy
- Lead advisory boards and external scientific forums to inform development and lifecycle decisions
Field Medical & Organizational Excellence
- Partner with Field Medical leadership to ensure high-quality, compliant scientific exchange
- Collaborate with Medical Excellence/Operations teams to strengthen governance, capabilities, training, and performance metrics across Medical Affairs
Leadership & Talent Development
- Lead and mentor senior medical professionals, fostering a culture of scientific rigor, accountability, and collaboration
- Contribute to the ongoing evolution of the Medical Affairs organization as the pipeline and global footprint continue to expand
Who You Are
You are an accomplished Medical Affairs leader with deep rare disease experience, strong scientific credibility, and the ability to operate effectively in highly complex environments. You are comfortable influencing at senior levels, navigating ambiguity, and balancing strategic vision with operational execution.
Qualifications
- Advanced degree required (MD, PharmD, PhD, or equivalent)
- 10+ years of Medical Affairs experience within biotechnology or pharmaceutical organizations
- Demonstrated leadership across rare disease portfolios, including experience with late-stage development and/or commercialization
- Strong background in evidence generation, scientific communications, and external engagement
- Proven ability to partner cross-functionally while maintaining Medical Affairs independence
- Prior people leadership experience strongly preferred
Why This Role Is Distinctive
- Opportunity to shape Medical Affairs strategy within a best-in-class rare disease organization
- High visibility role influencing long-term evidence, access, and patient impact
- Portfolio complexity that values thoughtful medical leadership over execution alone
- Collaborative, science-first culture with sustained investment in innovation
- Competitive compensation, long-term incentives, and comprehensive benefits
Working Model & Confidentiality
This role offers a hybrid working model with flexibility and meaningful collaboration. All discussions will be handled with the highest level of confidentiality.